AAVs in Retinal Gene Therapy
Type
Blog Post
...therapy had fallen - plummeted really - from grace 2 years prior with the death of Jesse Gelsinger in an... the whole field to a grinding stop and only now, 2 decades later, is it finally back in the limelight...the other hand, have broad tropism in the eye. AAV-2, the first identified serotype and the one used in...reading
1. FDA approves gene therapy for blindness
2. Gene Therapy emerges from disgrace to be the next...expression in non-dividing cells because they remain in episomal form in the cell’s nucleus.
But since nothing...hope are many more clinical successes for gene (and cell) therapies.
One may wonder why the first AAV-based... providing long-term gene expression in retinal cells - AAV - were becoming available.
At the same time...